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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT02469181
Other study ID # 4-2014-0679
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date May 21, 2015
Est. completion date October 2024

Study information

Verified date June 2021
Source Yonsei University
Contact Geu Ru Hong, M.D., Ph.D.
Phone 82-2-2228-8443
Email grhong@yuhs.ac
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

The purpose of this study is to evaluate the impact of ERT on LV diastolic function and flow in patients with Fabry's cardiomyopathy using diastolic stress echocardiography, LV vortex flow and CMR.


Description:

1. Objectives -The purpose of this study is to evaluate the impact of ERT on LV diastolic function and flow in patients with Fabry's cardiomyopathy using diastolic stress echocardiography, LV vortex flow and CMR. 2. Primary / Secondary Endpoint 1) Primary endpoint - Changes of peak exercise E/E' by diastolic stress echocardiography (RECAP-F STRESS trial) and LV vortex flow parameters (RECAP-F FLOW trial) at 1 year follow up. 2) Secondary endpoint - Changes of extracellular volume by CMR(T1 mapping) at 1 year follow up (RECAP-F trial) ② Evaluation of the degree of the resting LV diastolic function ③ Other echo-parameters; LV mass index at baseline, 1 year follow up, reduction of peak exercise E/E prime at 1 year follow up - Changes of quality of life using questionnaire ⑤ Change of peak VO2, exercise time, AT by diastolic stress echocardiography at 1 year follow up ⑥ Change in T1 baseline(myo, ms) & T1 baseline(blood, ms) T1 postcontrast(myo, ms) & T1 baseline(blood, ms) by CMR 3. Study Methods 1) Study Design : 28 patients with newly diagnosed genetically confirmed Anderson-Fabry's disease will undergo diastolic stress echocardiography, LV vortex flow analysis, and cardiac MRI before enzyme replacement therapy(ERT) (baseline study) and after 1 year of treatment with agalsidese beta at the dose of 1mg/kg (follow-up study). 2) Study procedures : Examinations as described below will be done before ERT and 1 year later


Recruitment information / eligibility

Status Recruiting
Enrollment 20
Est. completion date October 2024
Est. primary completion date October 2024
Accepts healthy volunteers No
Gender All
Age group 16 Years to 75 Years
Eligibility Inclusion Criteria: - Patients aged 16~75 years with Fabry's disease who were confirmed by enzyme assay and gene study - All patients should have LV hypertrophy in 2D echocardiography (end diastolic septum and posterior wall thickness =12mm) - Patients provided with the written, informed consent to participate in this study Exclusion Criteria: - Contraindication for agalsidase beta enzyme replacement treatment - Patients who cannot perform supine bicycle stress echocardiography, contrast echocardiography or cardiac MRI - Hemodynamically significant valvular heart disease or arrythmias - History of acute myocardial infarction or congestive heart failure with reduced LV ejection fraction of less than 35% - CVA in the prior 6 months - Scheduled or planned surgery in the next 6 months - Chronic liver cirrhosis - Allergy to contrast agent (Definity®, Lantheus Medical Imaging, North Billerica, MA, USA)

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
Korea, Republic of Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine Seoul

Sponsors (1)

Lead Sponsor Collaborator
Yonsei University

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type Measure Description Time frame Safety issue
Primary Changes of peak exercise E(velocity of early)/E'(Early diastolic) by diastolic stress echocardiography 1 year after the echocardiography
Secondary Change of peak VO2 by diastolic stress echocardiography 1 year after the echocardiography
Secondary Change of peak exercise time by diastolic stress echocardiography 1 year after the echocardiography
Secondary LV(left ventricular) vortex flow analysis by contract echocardiography 1 year after the echocardiography
Secondary Changes of extracellular volume by Cardiac MRI(CMR) 1 year after the CMR
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